Data from clinical trials will increasingly be subject to a “political crucible” as policymakers scrutinise more closely the cost-benefit value of publicly funded medicines, the editor of The Lancet warned last week.

Dr Richard Horton was summing up discussions at a meeting in London, organised by The Lancet and the James Lind Alliance, on how clinical trialists could more effectively serve the needs of clinicians and patients. He said the “very complex political environment” in which clinical data were now assessed was underlined by the judicial review of the National Institute for Health and Clinical Excellence’s decision to restrict UK NHS coverage of the Alzheimer’s disease drugs Aricept (donepezil), Exelon (rivastigmine) and Reminyl (galantamine).

As Horton pointed out, the then ongoing review had heard withering criticism of AD2000, a trial conducted by researchers from the University of Birmingham, with funding from the NHS Executive’s R&D Directorate, and published in The Lancet in June 2004. NICE’s Appraisal Committee has played down its reliance on AD2000 in its ruling on Eisai/Pfizer’s Aricept. The trial found that the drug did not significantly delay progression to more pronounced disability from Alzheimer’s diseaase or entry to institutional care, nor did it reduce the costs or burden of care for Alzheimer’s patients.

Validity of clinical evidence

Noting that the debate over Aricept and other Alzheimer’s drugs was not even about particularly expensive treatments, Horton said the demolition of the AD2000 data – which had come from a very reputable source – in the High Court raised questions about the validity of clinical evidence and how it was used in public policy. Scientists needed to be a “little bit more humble” about how their data were interpreted by policymakers, he suggested. Patients could help in this process by asking the kind of pertinent questions that would “flush out” data of real value.

As another speaker observed, though, patient involvement in shaping clinical decision-making can sometimes cloud objective judgment. Breast cancer charities had pushed en masse for rapid approval and NICE acceptance of Roche’s Herceptin (trastuzumab), backed up by emotionally charged media coverage suggesting the drug was a “cure-all,” noted general practitioner and Financial Times columnist Margaret McCartney. Less had been made, though, of Herceptin’s potential for cardiac side-effects or the fact that only 20%-30% of women contracted the HER2-positive breast cancer targeted by the drug.

McCartney was equally sceptical of what she saw as broad-brush government policies on more heavily treated conditions such as elevated cholesterol or hypertension. Citing the “vast amounts” of protocols on these diseases, she asked whether GPs were supposed to be treating individuals or populations and claimed that the government would be happy for doctors just to tick the relevant boxes and put all patients on drug therapy.

One delegate to the meeting protested that the discussions ignored an unassailable truth: most clinical research was funded by the pharmaceutical industry, whose sole responsibility was to its shareholders. Horton, however, was more optimistic. There seemed to be greater effort now in the UK to encourage and fund sources of independent clinical research – for example, through the Medical Research Council – as a counterweight to any industry agenda, he told PharmaTimes.